Duchenne muscular dystrophy

Muscular dystrophy drug launch halts after pricing concernsMarathon Pharmaceuticals is temporarily pausing the launch of its drug to treat a rare form of muscular dystrophy (MD) after concerns over its $89,000 price tag.
FDA approves first drug for Duchenne muscular dystrophyEven though an FDA panel recommended against approving a new drug to treat Duchenne muscular dystrophy in April, the agency granted accelerated approval to eteplirsen injection (Exondys 51, Sarepta Therapeutics) in mid-September.
Three drug trends impacting specialty pharmacyAMCP session, “Specialty Pharmaceutical in Development,” highlighted critical trends that healthcare executives should be watching
FDA drug approvals-April 2015FDA actions in brief, priority review, breakthrough therapy designation, fast-track designations, orphan drug designations, first-time generic approvals
CEO resignation linked to FDA drug approval processSarepta Therapeutics CEO Chris Garabedian resigned earlier this week, a move that may be linked to a dispute with the Food and Drug Administration (FDA) over the approval process for its new drug, according to The Wall Street Journal.
FDA drug approvals-January 2015FDA actions in brief, recommendations for approval, fast-track designations.
Early clues to muscular dystrophy in boysA combination of 2 developmental delays in toddler boys can alert physicians to the possibility of Duchenne muscular dystrophy and lead to earlier diagnosis, a new study reports.
FDA drug approvals-July 2014FDA drug approvals, priority review, breakthrough designation, fast-track designations, orphan drug designations, first-time generic approval
Nicox to refocus DMD treatmentNicox S.A. has decided to re-focus its naproxcinod development efforts, granting an undisclosed financial partner the right to enter into a period of exclusive evaluation to assess the potential development of the drug and of next-generation nitric oxide (NO)-donors outside the ophthalmology area.